The primary investigator of RESPOND discussed the trial’s goals and measures, as well as insight into why clinical trials like it are important in spinal muscular atrophy.
“I think this really is mimicking real-life experience, where we do have patients who have not really responded clinically to a single therapy for SMA. I do think that it’s really, really important to have clinical trial data to be able to look at outcomes.”
Biogen recently announced that the first patient was treated in the global phase 4 RESPOND study (NCT04488133) of nusinersen (Spinraza) in infants and children with spinal muscular atrophy (SMA). The trial’s goal is to assess the safety and efficacy of the gene therapy in patients who have unmet clinical needs despite treatment with another FDA-approved medicine, Zolgensma (onasemnogene abeparvovec; Avexis).
To find out more about the trial, NeurologyLive spoke with RESPOND primary investigator Julie Parsons, MD, Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders, professor of clinical pediatrics and neurology, at Children’s Hospital Colorado and the University of Colorado School of Medicine. She offered insight into what motivated the group to conduct the trial, and what she and her coinvestigators are hoping to observe. Particularly, she noted, they’re seeking the opportunity to confirm the utility of a different mechanism of action to improve clinical outcomes in patients with SMA.
In addition to detailing the measures of the study—including a number of relatively new outcomes—Parsons shared her insight into why clinical trials like this are so vitally important for this patient population.